PGN-EDO53, an experimental therapy designed to treat Duchenne muscular dystrophy (DMD) in patients amenable to exon 53 skipping, showed promising effects in preclinical studies, the therapy’s developer PepGen announced. The company also announced promising results from cell experiments for two other investigational exon-skipping therapies, PGN-EDO45 and PGN-EDO44.
I have three sons with Duchenne muscular dystrophy (DMD). We had little need for durable medical equipment when they were young, but their needs have changed as they’ve aged and their disease has progressed. We have hospital beds, power wheelchairs, a stair lift, and a ceiling lift system.
Sarepta Therapeutics plans to begin a clinical trial to test the antibody-cleaving therapy imlifidase as a pre-treatment for SRP-9001 (delandistrogene moxeparvovec) — its experimental gene therapy for Duchenne muscular dystrophy (DMD) — in patients with pre-existing antibodies against the gene therapy’s viral carrier. Preclinical work conducted by…
I celebrated my 27th birthday on Oct. 25 and feel tremendously blessed to have made it this far, especially with Duchenne muscular dystrophy. Duke (my pet name for Duchenne) has taken me through many arduous trials and difficult times, including feeling marginalized within my own disability community. Still,…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to Dyne Therapeutics’ DYNE-251 for the treatment of people with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 51 skipping. Fast track status is intended to accelerate a therapy’s development and expedite…
“We rejoice in our sufferings, knowing that suffering produces endurance, and endurance produces character, and character produces hope, and hope does not put us to shame, because God’s love has been poured into our hearts through the Holy Spirit who has been given to us.” I nervously recited this Bible…
Bleep. Bleep. Fire. Fire. Bleep. Bleep. It was 3 a.m., and my eyes flashed open as I bolted out of bed. The fire alarm was going off. Before I had left the bedroom, my 8-year-old daughter, Mary, was flying up the stairs from her basement bedroom, frantic and in tears.
Santhera and ReveraGen have completed their rolling application asking the U.S. Food and Drug Administration (FDA) to approve the dissociative corticosteroid vamorolone to treat Duchenne muscular dystrophy (DMD). Rolling applications allow companies to submit individual sections of the application as soon as they are completed, rather than waiting…
As a caregiver to my three sons living with Duchenne muscular dystrophy (DMD), I’ve had a few bad days lately. I’ve generally felt worn out and a little sad, and the parties around here have been pity parties. I try to be positive and remain in a place of…
Parent Project Muscular Dystrophy (PPMD) and Duchenne UK will jointly award up to $1 million to two research projects that seek to develop new treatments for Duchenne muscular dystrophy (DMD), especially those involving cell and gene therapy. The call for proposals for projects, which are expected…
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